Oklahoma City's Selexys Pharmaceuticals biopharmaceutical company announced Monday that the drug it has developed to treat sickle cell anemia was successful in its first phase of human testing.

Published: Wednesday, December 21, 2011 6:00 amBy:
Paula Burkes

Oklahoma City's Selexys Pharmaceuticals biopharmaceutical company announced Monday that the drug it has developed to treat sickle cell anemia was successful in its first phase of human testing.

A single dose, at 5 milligrams per kilogram of body weight, was found to prevent for up to one month the blocking of red blood cells  the genetic mutation that occurs in sickle cell patients, causing painful crises that can lead to heart attack, stroke and lung blockage.

So far, we've seen no side effects or reaction to the drug in patients, said Scott Rollins, Selexys president and chief executive. It really looks good.

Done in November, the trial involved 27 healthy volunteers of various ages and races, whose blood periodically was tested after they were given a placebo or various doses of the drug. Phase II will include 100 patients with the disease, Rollins said.

About 90,000 Americans, mostly blacks, have sickle cell, he said. The average age of survival is 40, and about 70 percent go untreated. The only applied therapy is several daily pills of an old, toxic chemotherapy drug that causes overproduction of one of the two types of hemoglobin, but only about 30 percent of patients respond and tolerate it, he said.

The prospective treatment  a mouse antibody genetically engineered to look like a human antibody  was discovered by Rodger McEver, head of cardiovascular biology at Oklahoma Medical Research Foundation, and manufactured by Cytovance Biologics in Oklahoma City.

The drug has been given orphan drug designation by the Food and Drug Administration for its potentially safe and effective treatment of a rare disorder. That means when the drug is publicly launched  which Rollins guesses will be in five years  Selexys will be awarded exclusive sales rights for 10 years.

Cytovance President Darren Head said his company is strongly committed to Selexys. It's an outstanding medical program, and we're excited to work on such an unmet need such as sickle cell treatment, he said. It took 18 months to develop the drug, Head said.

Selexys has $18 million in grant and venture capital money for the sickle cell drug.